Shots:The US FDA received BLA, plus a request for a priority review of apitegromab to improve motor function in pts with SMA who are on SMN-targeted therapy. The EMA’s MAA to be filed in Q1’25
The BLA was supported by the P-III (SAPPHIRE) & P-II (TOPAZ) trial, where SAPPHIRE met its 1EP of…
Shots: Dr. Jay Backstrom in an engaging conversation with PharmaShots, shares the highlights of the P-II Topaz trial designed to evaluate the safety and efficacy of apitegromab for the treatment of Spinal Muscle Atrophy (Type 2 & 3) With the potential to transform care in SMA by directly addressing progressive muscle weakness, apitegromab aims to…
Shots:Nagesh talked about how Spinal Muscular Atrophy (SMA) is affecting people all over the world and how Scholar Rock’s treatment options are improving the lives of those living with this diseaseNagesh also spoke about Scholar Rock’s lead product in SMA, apitegromab, highlighting its trial designs (TOPAZ & SAPPHIRE trials), outcomes, and real-world data…
